360.3 🏥 內科專科考前版

360.3.1 Mechanistic Deep Dive

360.3.1.1 HBV → HCC

  • HBV integration into host genome
  • Chronic inflammation
  • Cirrhosis pathway
  • HBx protein oncogenic
  • Can occur without cirrhosis (HBV unique)

360.3.1.2 MASLD/MASH → HCC

  • Metabolic stress
  • Inflammation
  • Fibrosis → cirrhosis → HCC
  • Can occur without cirrhosis in some
  • Rising globally

360.3.1.3 Atezolizumab + Bevacizumab Mechanism

  • Atezolizumab: anti-PD-L1
  • Bevacizumab: anti-VEGF (anti-angiogenic + immune microenvironment)
  • Synergistic
  • EGD before bev (variceal bleeding risk)

360.3.1.4 Durvalumab + Tremelimumab (STRIDE)

  • Single priming dose tremelimumab (anti-CTLA-4)
  • Followed by durvalumab (anti-PD-L1) monotherapy
  • HIMALAYA trial
  • No bevacizumab (alternative for varices/CV risk)

360.3.1.5 FGFR2 Fusion (iCCA)

  • 10-15% intrahepatic CCA
  • Pemigatinib, futibatinib, infigratinib
  • Activating fusions
  • Side effect: hyperphosphatemia, retinal detachment

360.3.1.6 IDH1 Mutation (iCCA)

  • 15-20% intrahepatic CCA
  • Ivosidenib FDA 2021
  • 2-hydroxyglutarate accumulation
  • ClarIDHy trial

360.3.2 Recent Trials & Updates

360.3.2.1 IMbrave150 (NEJM 2020)

  • Atezo + bev vs sorafenib
  • OS 19.2 vs 13.4 mo
  • New first-line HCC standard

360.3.2.2 HIMALAYA (NEJM 2022)

  • STRIDE (single tremelimumab priming + durvalumab)
  • vs sorafenib
  • OS 16.4 vs 13.8 mo
  • Alternative first-line

360.3.2.3 TOPAZ-1 (NEJM 2022)

  • Durva + cis/gem vs cis/gem
  • OS 12.8 vs 11.5 mo
  • New first-line CCA

360.3.2.4 KEYNOTE-966 (Lancet 2023)

  • Pembro + cis/gem
  • Similar to TOPAZ-1
  • Confirmation

360.3.2.5 LEAP-002 (Failed)

  • Pembrolizumab + lenvatinib
  • Did not beat lenvatinib alone

360.3.2.6 CheckMate-9DW (2024)

  • Nivolumab + ipilimumab vs lenvatinib/sorafenib
  • Positive OS
  • Potential new first-line

360.3.2.7 MERIT-12 / IMbrave050 (Adjuvant)

  • Adjuvant atezo + bev for high-risk resected HCC
  • Mixed results

360.3.3 High-Yield Specialist Points

360.3.3.1 AFP Limitations

  • Not specific (can ↑ in cirrhosis, hepatitis)
  • Not sensitive (~ 50% miss)
  • Use with US
  • AFP > 400 with cirrhosis suggestive
  • DCP (PIVKA-II) emerging

360.3.3.2 LI-RADS Categories

  • LR-1 (definitely benign)
  • LR-2 (probably benign)
  • LR-3 (intermediate)
  • LR-4 (probably HCC)
  • LR-5 (definitely HCC) — no biopsy needed
  • LR-M (probably/definitely malignant, not HCC)
  • LR-TIV (tumor in vein)

360.3.3.3 Bridging/Downstaging

  • TACE, TARE, ablation while awaiting transplant
  • Downstaging to bring within Milan criteria
  • UCSF criteria extended (≀ 6.5 cm single or ≀ 3 ≀ 4.5 cm total ≀ 8 cm)

360.3.3.4 Combined Hepatocellular-Cholangiocarcinoma

  • Mixed phenotype
  • Worse prognosis
  • Generally not transplant candidate

360.3.3.5 Fibrolamellar HCC

  • Young adults (15-30)
  • Non-cirrhotic liver
  • No HBV/HCV association
  • DNAJB1-PRKACA fusion
  • Resection is mainstay

360.3.3.6 Cholangiocarcinoma Surgical Approach

  • iCCA: hepatic resection
  • pCCA (Klatskin): hepatectomy + bile duct excision + Roux-en-Y
  • dCCA: Whipple
  • Mayo protocol LT for hilar CCA

360.3.3.7 Lynch Syndrome and CCA

  • Increased risk
  • Family history important

360.3.3.8 Cholangioscopy

  • SpyGlass
  • Direct visualization
  • Biopsy targeted

360.3.3.9 Photodynamic Therapy (PDT)

  • Palliative for unresectable CCA
  • Endoscopic
  • Limited use

360.3.3.10 NET WHO Classification

  • G1: Ki-67 < 3%
  • G2: Ki-67 3-20%
  • G3: Ki-67 > 20%
  • NEC (neuroendocrine carcinoma) — poorly differentiated
  • Different treatment approach (chemo)

360.3.3.11 MENA1 + NETs

  • Pancreatic NETs (insulinoma, gastrinoma, others)
  • Multiple endocrine neoplasia type 1
  • RET/MEN2 = MTC

360.3.4 Pearls

  • HCC surveillance: US ± AFP q6 mo
  • LI-RADS LR-5 = HCC (no biopsy)
  • BCLC integrates everything
  • IMbrave150 = atezo + bev (first-line)
  • HIMALAYA = STRIDE durva + treme
  • Milan Criteria for transplant
  • TOPAZ-1 = durva + cis/gem (CCA first-line)
  • Pemigatinib (FGFR2), ivosidenib (IDH1) CCA
  • Mayo Protocol for hilar CCA + LT
  • GIST: imatinib (KIT/PDGFRA)